Trials / Terminated
TerminatedNCT02177825
Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas
Phase II Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas
- Status
- Terminated
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 5 (actual)
- Sponsor
- St. Justine's Hospital · Academic / Other
- Sex
- All
- Age
- 2 Years – 21 Years
- Healthy volunteers
- Not accepted
Summary
This phase II trial will test the hypothesis that inhibition of c-kit signalling pathways in pediatric patients with Neurofibromatosis Type I(NF-1) and progressing plexiform neurofibroma will result in objective reduction and/or inhibition of plexiform neurofibromas progression. This will be a Phase II study of imatinib mesylate given orally. Patients with stable or responding disease may receive the drug for a period not exceeding one year.
Detailed description
Clinical objectives 1. Demonstrate the clinical benefit of imatinib in a pediatric patient population with progressing and metabolically active plexiform neurofibromas (NF) 2. Demonstrate the need or not to pursue treatment for more than a year in responders to imatinib Biological studies objectives 1. Identify biological markers of plexiform neurofibroma progression and response to treatment 2. Identify biological markers of mast cell responses to imatinib, given that mast cells are required for tumorigenesis and are a target for imatinib Imaging studies objectives Using 18-Fluorodeoxyglucose-positron Emission Tomography (FDG PET/CT): 1. Identify imaging characteristics of progressing plexiform neurofibromas 2. Assess the role of F18-FDG PET/CT in comparison with CT/MRI to evaluate response to imatinib ¸ Pharmacological study 1. Evaluate trough plasma levels of imatinib and its active metabolite (NDMIL N-desmethyl imatinib) achieved in this pediatric population 2. Identify potential correlation between imatinib (and NDMI) trough levels achieved and clinical response
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Imatinib Mesylate | oral administration |
Timeline
- Start date
- 2014-06-01
- Primary completion
- 2019-03-01
- Completion
- 2019-03-01
- First posted
- 2014-06-30
- Last updated
- 2019-04-08
Locations
1 site across 1 country: Canada
Source: ClinicalTrials.gov record NCT02177825. Inclusion in this directory is not an endorsement.